PGEN - Precigen Is Pioneering a Nonsurgical Treatment for a Debilitating Respiratory Disease; Positive Data Released on Recurrent Respiratory Papillomatosis Showed a 50% Complete Response
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New hope may be onthe horizon for sufferers of Recurrent Respiratory Papillomatosis, orRRP, a rare and debilitating disease that typically requires patientsto undergo multiple surgeries each year to remove benign tumors calledpapillomas, that grow in the respiratory tract.
Precigen, Inc.(Nasdaq: PGEN ) released complete data at a virtual R&DDay event on January 24, 2023 at 4:30 PM ET from a robust, openlabel Phase 1 trial of its investigational candidate PRGN-2012, aninnovative gene-based immunotherapy designed to inhibit or prevent thegrowth of the dreaded papillomas in the hopes of reducing the numberof surgeries.
Clinical datapresented showed a strong response in RRP patients with 50% ofpatients in complete response, requiring no post-treatment surgeries,following PRGN-2012 treatment at Dose Level 2. All completeresponders remained surgery-free post-treatment with a minimum12-months of follow up. PRGN-2012 treatment at Dose Level 2significantly reduced the need for surgeries in severe, aggressive RRPpatients with the median number of surgeries in 12-month period beingreduced from 6.5 pre-treatment to 0.5 post-treatment. The data alsoshowed that PRGN-2012 was very well tolerated by patients, with nodose-limiting toxicities or treatment-related adverse events aboveGrade 2.
A Phase 2 trial ofPRGN-2012 in RRP is also underway and, depending on the results, itmay qualify as a pivotal trial to facilitate the filing of a BiologicsLicense Applications (BLA). It is approximately 85 percent enrolled todate.
RRP affects bothchildren and adults. Symptoms include a hoarse voice, difficultysleeping and swallowing, chronic coughing, and breathing problems. Inextreme cases, the disease can be fatal because the papillomas causesevere airway obstruction to the lungs and the lungs become floodedwith fluids and infection. In a minority of patients RRP canmalignantly transform into cancer.
The company estimates indicate there are up to 16,000 activecases of RRP in the US and as many as 75,000 total worldwide, althoughthere are believed to be thousands more undiagnosed cases. Notherapeutic treatments are currently approved by the FDA. Companyestimates suggest PRGN-2012 could have peak annual US sales ofapproximately $1 billion if approved.
There is a high need for safe and effective therapeutic optionsfor RRP. Currently,the only standard-of-care treatment for RRP is repeat surgeries tomaintain voice and respiratory function. The sad reality is thatmany RRP patients require hundreds of surgeries over their lifetimes.The quality of life and economic burden of recurrent surgeries onpatients, their families and physicians is very high.
The surgeries are onlyeffective at removing the built-up Human Papillomavirus Virus(HPV)-infected lesions in the respiratory tract, but they do notprevent further buildup. Patients often struggle to maintainconsistent employment because of the frequent surgeries all whileincurring costly medical bills.
Moreover,frequent surgeries for RRP can worsen a patient’s condition overtimeby potentially increasing the spread of the HPV virus and thelikelihood of serious co-morbidities, including breathing issues andloss of vocal function.
PRGN-2012Seen as Promising, Potentially Less Costly Solution
Lacking other treatmentsto potentially reduce the frequency of surgeries, some physicians haveturned to off-label use of drugs approved for other indications, butwithout proven benefit for RRP patients.
One such drug is cidofovir, a broad-spectrum antiviral drugfrom Gilead (GILD) and Pfizer (PFE) that wasoriginally approved for treating CMV and HIV infection. It isprescribed by some physicians treating RRP, but it can cause livertoxicity and patients typically rebound after withdrawal, making it–at best-- a short-term solution.
Another is Avastin® from Roche Holdings (RHHBY) ,an antiangiogenic drug in wide use as a cancer therapy. Avastin hasbeen shown to temporarily slowdown papilloma growth in some RRPpatients. However, it can cause severe side effects and cannotactivate specific T cells needed to eliminate HPV infected cellsresponsible for papilloma growth in RRP.
Currently, the most promising solution appears to bePrecigen’s PRGN-2012 gene therapy which works by activating anti-HPVT cells to induce immunity against HPV subtypes 6 and 11, which areresponsible for RRP.
If theon-going Phase 2 trial shows positive results, PRGN-2012 could be onits way to becoming a standard of care for reducing the burden ofsurgeries, lowering care costs and improving the quality of life ofRRP patients.
In a studyconducted on behalf of Wells Fargo, 10 ear, nose, throat physicianswho treat RRP responded very favorably to the use of a drug with aPRGN-2012-target profile of safety and efficacy.
Precigen’sclinical-stage pipeline also includes Phase 1 and Phase 2 programs inimmuno-oncology and infectious and autoimmune diseases. PRGN-2012 is Precigen’sinnovative therapeutic vaccine for RRP patients.
Analyst price targetsfor PGEN as of August 2022 range from $6 to $14, with buy or marketoutperform ratings issued by Stifel, JMP Securities, CantorFitzgerald, and H.C. Wainwright.
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Precigen: Advancing Medicine withPrecision™Precigen (Nasdaq: PGEN) is a dedicated discovery andclinical stage biopharmaceutical company advancing the next generationof gene and cell therapies using precision technology to target themost urgent and intractable diseases in our core therapeutic areas ofimmuno-oncology, autoimmune disorders,and infectious diseases. Ourtechnologiesenable us to find innovative solutions for affordablebiotherapeutics in a controlled manner. Precigen operates as aninnovation engine progressing a preclinical and clinical pipeline ofwell-differentiated therapies toward clinical proof-of-concept andcommercialization. For more information about Precigen, visitwww.precigen.com or follow us on Twitter @Precigen, LinkedIn orYouTube.
This post contains sponsored advertising content. Thiscontent is for informational purposes only and is not intended to beinvesting advice.
Some of the statements made in this pressrelease are forward-looking statements. These forward-lookingstatements are based upon the Company's current expectations andprojections about future events and generally relate to plans,objectives, and expectations for the development of the Company'sbusiness, including the timing and progress of preclinical studies,clinical trials, discovery programs and related milestones, thepromise of the Company's portfolio of therapies, and in particularits CAR-T and AdenoVerse therapies. Although management believes thatthe plans and objectives reflected in or suggested by theseforward-looking statements are reasonable, all forward-lookingstatements involve risks and uncertainties, including the possibilitythat the timeline for the Company's clinical trials might beimpacted by the COVID-19 pandemic, and actual future results may bematerially different from the plans, objectives and expectationsexpressed in this press release. The Company has no obligation toprovide any updates to these forward-looking statements even if itsexpectations change. All forward-looking statements are expresslyqualified in their entirety by this cautionary statement. For furtherinformation on potential risks and uncertainties, and other importantfactors, any of which could cause the Company's actual results todiffer from those contained in the forward-looking statements, see thesection entitled "Risk Factors" in the Company's mostrecent Annual Report on Form 10-K and subsequent reports filed withthe Securities and Exchange Commission.
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